Exploring a Splice Modulation Antisense Oligonucleotide to Restore STATHMIN-2 Levels in ALS Patients

Time: 11:00 am
day: Day Two AM


• A splice modulation ASO strategy with the aim to treat 90% of the total ALS population (TDP-43 positive patients)

• Optimizing dosage to ensure efficacious and safe therapeutic output

• Biomarker strategies to evaluate target engagement and efficacy for STMN2 ASO trials